Cell Therapy in HFpEF

Phase 2

Completed

• Conditions: Heart Failure With Normal Ejection Fraction

• Registration Number: NCT02923609
• Lead Sponsor: University Medical Centre Ljubljana

Brief Summary

The primary objective of the study is to investigate safety and efficacy of transendocardial CD34+ cell therapy in patients with HFpEF by evaluating changes in myocardial structure and function, patient exercise capacity and clinical outcome.

The safety end-points include serious adverse events (SAEs), defined as any serious event that may result in persistent or significant disability or incapacity and included death, heart transplantation, sustained ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation), and heart failure exacerbation requiring hospitalization.

Detailed Description

In all patients, peripheral blood stem cells will be mobilized by daily subcutaneous injections of G-CSF (10 mcg/kg, divided b.i.d) for 5 days. Peripheral blood stem cells will then be collected with Miltenyi cell separator (Miltenyi Biotech, Germany) and the magnetic cell separator Isolex 300i (Nexell Therapeutics Inc., California, USA) will be used for the immunomagnetic positive selection of the CD34+ cells.

Study Timeline

• Study Start: 2016-01
• Study First Submitted: 2016-10-03
• Study First Submitted QC: 2016-10-03
• Study First Posted: 2016-10-04
• Primary Completion: 2022-01
• Study Completion: 2022-03
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-09
• Last Update Posted: 2023-05-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Preserved left ventricular systolic function on echocardiography (LVEF>50%)
• Evidence of diastolic dysfunction by echocardiography (E/e'>15)
• Symptoms of heart failure
• NT-proBNP levels >300 pg/ml
• absence of permanent atrial fibrillation

Exclusion Criteria

• acute multi-organ failure
• history of any malignant disease within 5 years
• diminished functional capacity due to non-cardiac co-morbidities (COPD, PAOD, morbid obesity)
• pregnancy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Change in left ventricular filling pressures (E/e') assessed by echocardiography	Baseline, 6 months and 1 year	The E/e' ratio will be calculated from early transmitral velocity divided by peak left ventricular relaxation velocity for estimation of the left ventricular filling pressure.

Secondary Outcome Measures

Name	Time	Method
Change in diastolic dysfunction grade	Baseline, 6 months and 1 year	Diastolic dysfunction will be graded according based on E/A ratio and left atrial pressure estimation.
Change in exercise capacity	Baseline, 6 months and 1 year	6-minute walk test will be performed by a blinded observer according to the standard protocol.
Change in NT-proBNP levels	Baseline, 6 months and 1 year	NT-proBNP assays will be performed at a central independent laboratory, blinded to the patient's clinical data using a commercially available kit (Roche Diagnostics, Mannheim, Germany).
Change in systolic strain	Baseline, 6 months and 1 year	Left ventricular longitudinal strains will be analyzed by speckle tracking echocardiography from apical four-chamber, two-chamber, and long-axis views.

Trial Locations

Locations (1)

University Medical Center Ljubljana; 🇸🇮 Ljubljana, Slovenia